Charcot-Marie-Tooth Disease - Pipeline Review, H2 2016

 Table of Contents

Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Charcot-Marie-Tooth Disease Overview 7
Therapeutics Development 8
Pipeline Products for Charcot-Marie-Tooth Disease - Overview 8
Pipeline Products for Charcot-Marie-Tooth Disease - Comparative Analysis 9
Charcot-Marie-Tooth Disease - Therapeutics under Development by Companies 10
Charcot-Marie-Tooth Disease - Therapeutics under Investigation by Universities/Institutes 11
Charcot-Marie-Tooth Disease - Pipeline Products Glance 12
Late Stage Products 12
Early Stage Products 13
Charcot-Marie-Tooth Disease - Products under Development by Companies 14
Charcot-Marie-Tooth Disease - Products under Investigation by Universities/Institutes 15
Charcot-Marie-Tooth Disease - Companies Involved in Therapeutics Development 16
Acetylon Pharmaceuticals, Inc. 16
Addex Therapeutics Ltd 17
Affectis Pharmaceuticals AG 18
Genzyme Corporation 19
InFlectis BioScience 20
Lead Discovery Center GmbH 21
PharmatrophiX, Inc. 22
Pharnext SAS 23
Charcot-Marie-Tooth Disease - Therapeutics Assessment 24
Assessment by Monotherapy Products 24
Assessment by Combination Products 25
Assessment by Target 26
Assessment by Mechanism of Action 28
Assessment by Route of Administration 30
Assessment by Molecule Type 31
Drug Profiles 33
(baclofen + naltrexone + sorbitol) - Drug Profile 33
Product Description 33
Mechanism Of Action 33
R&D Progress 33
ADX-71441 - Drug Profile 35
Product Description 35
Mechanism Of Action 35
R&D Progress 35
AFC-5128 - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
IFB-088 - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
LM-22B10 - Drug Profile 40
Product Description 40
Mechanism Of Action 40
R&D Progress 40
Small Molecule to Inhibit HDAC6 for Charcot-Marie-Tooth Disease - Drug Profile 41
Product Description 41
Mechanism Of Action 41
R&D Progress 41
Small Molecule to Inhibit Rab7 GTPase for Oncology, Neuropathy, Immunology, Neurodegenerative Diseases, Lipidoses and Charcot-Marie-Tooth Disease Type II - Drug Profile 42
Product Description 42
Mechanism Of Action 42
R&D Progress 42
Small Molecules for Charcot-Marie-Tooth Disease Type I A - Drug Profile 43
Product Description 43
Mechanism Of Action 43
R&D Progress 43
Small Molecules to Inhibit HDAC-6 for Immunology, Oncology and Genetic Disorders - Drug Profile 44
Product Description 44
Mechanism Of Action 44
R&D Progress 44
Small Molecules to Inhibit PMP22 for Charcot-Marie-Tooth Type 1 A - Drug Profile 45
Product Description 45
Mechanism Of Action 45
R&D Progress 45
Stem Cell Therapy to Activate PMP22 for Charcot-Marie-Tooth Disease Type 1A - Drug Profile 46
Product Description 46
Mechanism Of Action 46
R&D Progress 46
Charcot-Marie-Tooth Disease - Dormant Projects 47
Charcot-Marie-Tooth Disease - Product Development Milestones 48
Featured News & Press Releases 48
Apr 25, 2016: Pharnext Announces the Opening of the First U.S. Study Site for its PLEO-CMT International Pivotal Phase 3 Trial in Charcot-Marie-Tooth Disease Type 1A 48
Feb 16, 2016: Pharnext Supports the Fight Against Rare Diseases through Development of Pleotherapy-based Treatments 49
Jan 25, 2016: IFB-088 (Sephin1) receives orphan drug designation in the European Union for the treatment of Charcot-Marie-Tooth disease 49
Dec 15, 2015: Pharnext Initiates PLEO-CMT, an International Pivotal Phase 3 Trial, in Charcot-Marie-Tooth disease type 1A 50
Oct 27, 2015: FDA grants orphan drug designation to IFB-088 (Sephin1) for the treatment of Charcot-Marie-Tooth disease 51
Jun 30, 2015: Pharnext's PXT-3003 Featured in Multiple Presentations at 2015 Peripheral Nerve Society Biennial Meeting 51
Jan 13, 2015: InFlectis BioScience banks €1.75m to realise the potential of IFB-088 drug candidate, a breakthrough therapeutic approach to treat neurodegenerative diseases 52
Dec 17, 2014: Pharnext Announces Pleotherapy Proof of Concept in Charcot-Marie-Tooth Disease Type 1A through Two Peer-Reviewed Publications 52
Oct 21, 2014: Addex And The Charcot-Marie-Tooth Association Enter Collaboration To Advance ADX71441 In Charcot-Marie-Tooth 1A disorder 53
Jun 23, 2014: The EMA and FDA Grant Orphan Drug Designation to Pharnext's PXT-3003 for the Treatment of Charcot-Marie-Tooth Disease Type 1A 54
Oct 03, 2013: Addex ADX71441 Dose Dependently Reduced PMP22 Expression Comparable to Baclofen in a Pre-Clinical Transgenic Model of Charcot-Marie-Tooth 1A Disease 55
Apr 24, 2013: Addex Receives Approval To Initiate Phase I First-In-man Clinical Study For ADX71441 55
Jan 22, 2013: Addex Receives Broad US Composition Of Matter Patent For ADX71441 56
Jan 07, 2013: Addex Therapeutics Announces Positive Data With ADX71441 In Pre-Clinical Transgenic Model Of Charcot-Marie-Tooth 1A Disease 57
Nov 22, 2011: Pharnext Completes Patient Recruitment For Phase II Study Of PXT3003 In Type 1A Charcot-Marie-Tooth Disease 57
Appendix 59
Methodology 59
Coverage 59
Secondary Research 59
Primary Research 59
Expert Panel Validation 59
Contact Us 59
Disclaimer 60

List of Tables

Number of Products under Development for Charcot-Marie-Tooth Disease, H2 2016 8
Number of Products under Development for Charcot-Marie-Tooth Disease - Comparative Analysis, H2 2016 9
Number of Products under Development by Companies, H2 2016 10
Number of Products under Investigation by Universities/Institutes, H2 2016 11
Comparative Analysis by Late Stage Development, H2 2016 12
Comparative Analysis by Early Stage Development, H2 2016 13
Products under Development by Companies, H2 2016 14
Products under Investigation by Universities/Institutes, H2 2016 15
Charcot-Marie-Tooth Disease - Pipeline by Acetylon Pharmaceuticals, Inc., H2 2016 16
Charcot-Marie-Tooth Disease - Pipeline by Addex Therapeutics Ltd, H2 2016 17
Charcot-Marie-Tooth Disease - Pipeline by Affectis Pharmaceuticals AG, H2 2016 18
Charcot-Marie-Tooth Disease - Pipeline by Genzyme Corporation, H2 2016 19
Charcot-Marie-Tooth Disease - Pipeline by InFlectis BioScience, H2 2016 20
Charcot-Marie-Tooth Disease - Pipeline by Lead Discovery Center GmbH, H2 2016 21
Charcot-Marie-Tooth Disease - Pipeline by PharmatrophiX, Inc., H2 2016 22
Charcot-Marie-Tooth Disease - Pipeline by Pharnext SAS, H2 2016 23
Assessment by Monotherapy Products, H2 2016 24
Assessment by Combination Products, H2 2016 25
Number of Products by Stage and Target, H2 2016 27
Number of Products by Stage and Mechanism of Action, H2 2016 29
Number of Products by Stage and Route of Administration, H2 2016 30
Number of Products by Stage and Molecule Type, H2 2016 32
Charcot-Marie-Tooth Disease - Dormant Projects, H2 2016 47 

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